Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy


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Trial ID: NCT03179631


This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

Official Title

A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Stanford Investigator(s)

John W. Day, MD, PhD
John W. Day, MD, PhD

Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology


Inclusion Criteria:

   - Male sex

   - Age ≥5 years

   - Phenotypic evidence of Duchenne Muscular Dystrophy

   - Nonsense point mutation in the dystrophin gene

   - Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum
   of 12 months immediately prior to start of study treatment, with no significant change
   in dosage or dosing regimen for a minimum of 3 months immediately prior to start of
   study treatment

   - 6MWD ≥150 meters

   - Ability to perform timed function tests within 30 seconds

   - Willingness and ability to comply with scheduled visits, drug administration plan,
   study procedures, laboratory tests, and study restrictions.

Exclusion Criteria:

   - Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start
   of study treatment.

   - Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.

   - Prior or ongoing therapy with ataluren.

   - Known hypersensitivity to any of the ingredients or excipients of the study drug

   - Exposure to another investigational drug within 6 months prior to start of study
   treatment, or ongoing participation in any interventional clinical trial.

   - History of major surgical procedure within 12 weeks prior to start of study treatment,
   or expectation of major surgical procedure during the 72-week placebo-controlled
   treatment period.

   - Requirement for daytime ventilator assistance or any use of invasive mechanical
   ventilation via tracheostomy.

   - Uncontrolled clinical symptoms and signs of congestive heart failure

   - Elevated serum creatinine or cystatin C at screening.


drug: Ataluren



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Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Monica Sangco